Published on August 23, 2016
What are the main therapies for essential thrombocythemia (ET), and how do they help? Dr. John Mascarenhas, a myeloproliferative neoplasm (MPN) expert at Mount Sinai School of Medicine, explains the various treatments currently used for ET. Dr. Mascarenhas discusses the risk of thrombotic (blood clot) episodes that characterize this disease.
Transcript | The Goal of Essential Thrombocythemia (ET) Treatment
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Dr. Mascarenhas, when you have ET—we were just describing what that was—what do these drugs do, whether it’s low-dose aspirin or the other drugs to try to control ET? What are they doing?
It’s important to first ask the question what are we trying to accomplish when we treat ET?
Really the goal of treating ET is to try to reduce the risk of thrombosis, which can be a major complication, which can add to comorbidity mortality…
A stroke, a heart attack, a clot in the lungs or the legs. Basically a clot anywhere it can occur. Patients with ET, unfortunately, are at a heightened risk for this. So really the emphasis is on trying to reduce that risk. So aspirin is usually the cornerstone of risk reduction. And then for patients who we think are at a higher risk, whether it’s age or a history of thrombosis or cardiovascular risk factors that may be present, we usually employ cytoreductive therapy.
Earlier there was a question about what cytoreductive therapy is. It’s typically any drug that reduces blood counts, in the simplest terms. So hydroxyurea (Hydrea) is an example of a chemotherapeutic cytoreductive therapy. Anagrelide (Agrylin) is an example of a drug that targets platelet reductions specifically. So these are different drugs. Interferon is a drug, an immunomodulatory drug that affects the production of counts. These are drugs that really affect the production of countforemost and we believe therefore reduces the risk of thrombotic complications.
There are probably mechanisms that we don’t—not probably, there are definitely mechanisms that we don’t totally understand that these drugs probably also employ that further help the risk reduction, and that’s an area of ongoing research.
Are all of these, including interferon, a pill?
No. Anagrelide, hydroxyurea and obviously aspirin are oral. Interferon, whether it’s interferon or Pegasys, a longer-acting formulation, are injectables. These are subcutaneous injections that are given typically once a week.
Do people come to the clinic, or they do it at home?
They can do it at home, so it’s typically dispensed as syringes and a patient can self-inject and has the autonomy to decide when and where they’re going to do that.